Publication: RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
Issued Date
2009-01-27
Resource Type
ISSN
10916490
00278424
00278424
Other identifier(s)
2-s2.0-59049089272
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Mahidol University
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SCOPUS
Bibliographic Citation
Proceedings of the National Academy of Sciences of the United States of America. Vol.106, No.4 (2009), 1205-1210
Suggested Citation
Saovaros Svasti, Thipparat Suwanmanee, Suthat Fucharoen, Hong M. Moulton, Michelle H. Nelson, Nobuyo Maeda, Oliver Smithies, Ryszard Kole RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice. Proceedings of the National Academy of Sciences of the United States of America. Vol.106, No.4 (2009), 1205-1210. doi:10.1073/pnas.0812436106 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/28397
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Title
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
Abstract
Repair of β-globin pre-mRNA rendered defective by a thalassemia-causing splicing mutation, IVS2-654, in intron 2 of the human β-globin gene was accomplished in vivo in a mouse model of IVS2-654 thalassemia. This was effected by a systemically delivered splice-switching oligonucleotide (SSO), a morpholino oligomer conjugated to an arginine-rich peptide. The SSO blocked the aberrant splice site in the targeted pre-mRNA and forced the splicing machinery to reselect existing correct splice sites. Repaired β-globin mRNA restored significant amounts of hemoglobin in the peripheral blood of the IVS2-654 mouse, improving the number and quality of erythroid cells. © 2009 by The National Academy of Sciences of the USA.